OBJECTIVES

: Waldenström’s macroglobulinemia (WM) is a rare, lymphoproliferative B-cell disorder characterised by infiltration of lymphoplasmacytic cells into the bone marrow and immunoglobulin M monoclonal gammopathy. As an indolent sub-type of non-Hodgkin’s lymphoma, WM has a long survival time, with median overall survival time of between 5 and 10 years. Due to the disease’s rarity, there are no published estimates of the number of patients requiring a new line of therapy for WM across Europe.

METHODS

: A model was developed to estimate the number of patients in and across the EU28 with WM who required a new (LOT) treatment in a given year. Population data from the World Bank, epidemiological data from GLOBOCAN and the literature, and efficacy data from published and unpublished observational studies were used to populate the model. The model tracked incident patients from 2000 to 2017 and produced estimates of WM patient numbers between 2018 and 2022. Patients were assumed to be 72 years of age at diagnosis based on prior published literature.

RESULTS

: Incidence of WM was found to vary in the literature from 0.32 per 100,000 to 0.83 per 100,000. Across the EU28 over the 5-year period the model estimated that in total between 8,128 and 20,952 patients will require new first-line treatments, between 6,665 and 17,181 patients will require second-line treatments and between 5,771 and 14,876 third-line treatments. Of the new patients diagnosed in the next five years, between 2,066 and 5,325 will be asymptomatic at diagnosis.

CONCLUSIONS

: The model predicted considerable demand for second- and third-line therapies in WM. With the indolent nature of the disease, and large demand of new treatments in elderly patients, it is essential treatments with good tolerability be made available.